The first two patients in uniQure’s in-human gene therapy trial for Huntington’s disease (HD) have now been treated. The dosing of the drug called AMT-130 in the trial marks a significant milestone for uniQure and for Huntington patients in need of treatment options.
At this stage, safety, tolerance and efficacy of the treatment are being tested. 26 brave patients will participate in the first phase of the study. Here, 16 will get the gene therapy treatment, while 10 will undergo a so-called “imitation” surgery.
The participants will then be followed for 18 months. The initial results of the treatment’s efficacy and safety is therefore expected to be known in the Spring of 2022.
uniQure’s gene therapy is unique and different from other Huntington treatments being tested: it is a single treatment, it alter the genetic makeup and it is being carried out with human participants.